Base Editing as a Therapeutic Strategy for Autosomal Dominant Tubulointerstitial Kidney Disease caused by Pathogenic UMOD Variants
Nora Diederich, Sarah Wendlinger, Shuhan Kong, Dimitra Papaioannou, Emily Haar, Amr Al‐Shebel, Guy Yealland, Wenjun Jin, Carlos Cordero Oropeza, Ria Anne-Rose Schönauer, Roberta Di Costanzo, Marta Mariniello, Annika Fendler, Bernhard Ralla, Kai‐Uwe Eckardt, , Michael M. Kaminski
This paper evaluates base editing strategies for treating ADTKD-UMOD by correcting pathogenic UMOD variants and inactivating mutant alleles, showing efficient correction and expression modulation in cell models.
bioRxiv · Oct 16, 2026 · PreprintGenetics